RemeGen's Telitacicept (RC18) Received Orphan Drug Designation from EMA for Myasthenia Gravis

Rhea-AI Summary

RemeGen announced that telitacicept (RC18) received Orphan Drug Designation (ODD) from the European Medicines Agency for treating Myasthenia Gravis (MG), becoming the first dual-target biologic drug for MG with ODD from both FDA and EMA globally. The designation provides benefits including protocol assistance, fee reductions, and 10 years of market exclusivity. Telitacicept, already approved in China in May 2025, showed impressive Phase III results with 98.1% of participants showing ≥3 points improvement in MG-ADL (vs 12% placebo) and 87% showing ≥5 points improvement in QMG score (vs 16% placebo). The drug works by blocking BLyS and APRIL signaling pathways to inhibit abnormal B cells and reduce pathogenic autoantibodies. MG affects 15-25 per 100,000 people globally, and current treatments often face challenges with poor response and disease relapse.

Positive

• First dual-target biologic drug for MG to receive ODD from both FDA and EMA worldwide
• Impressive Phase III results with 98.1% efficacy vs 12% placebo in MG-ADL improvement
• Secured 10 years of market exclusivity in Europe through ODD designation
• Already approved for marketing in China as of May 2025

Negative

• None.

YANTAI, China, June 17, 2025 /PRNewswire/ -- On June 16, 2025, RemeGen Co., Ltd. ("RemeGen", stock symbols: 688331.SH/09995.HK) announced that telitacicept (RC18; brand name: 泰爱^®) has received Orphan Drug Designation (ODD) from European Medicines Agency (EMA) for the treatment of Myasthenia Gravis (MG), marking a key milestone achieved in its global development. Telitacicept now is the first dual-target biologic drug for MG with ODD from both FDA and EMA worldwide.

The designation, granted based on telitacicept's significant benefits in treating the life-threatening rare disease of MG, will provide advantages to this novel agent on protocol assistance, regulatory fee reductions/waivers and up to 10 years of market exclusivity, thereby accelerating the clinical development, registration and drug approval process in Europe and its availability among MG patients.

MG is an acquired antibody-mediated rare autoimmune disorder affecting the neuromuscular junction. According to the Myasthenia Gravis Foundation of America (MGFA) and various studies, its global prevalence is estimated to be 15-25 per 100,000, meeting EMA's definition of the rare disease (the prevalence of less than 5 in 10,000 in the EU). Though the current treatment options (including cholinesterase inhibitors, glucocorticoids, immunosuppressants, intravenous immunoglobulins, plasma exchange, and targeted biological agents) may help to manage symptoms, many patients still suffer from poor response, drug intolerance and disease relapse, highlighting a significant unmet clinical need.

Telitacicept is the world's first approved innovative BLyS/APRIL dual-targeting fusion protein drug for MG. The pathology of MG is characterized by the autoantibodies produced by pathological B cells attacking the neuromuscular junction protein (such as acetylcholine receptor, muscle-specific tyrosine kinase). Telitacicept can block both BLyS and APRIL signaling pathways, effectively inhibiting abnormally activated B cells and reducing the production of pathogenic autoantibodies, which is expected to interfere with the source of disease progress of MG.

Telitacicept was approved for marketing in China in May this year. Its phase III clinical trial boasts excellent results: after treatment with telitacicept for 24 weeks, improvement ≥ 3 points in myasthenia gravis-activities of daily living (MG-ADL) was observed in 98.1% of participants (12.0% in the placebo group) and improvements ≥ 5 points in quantitative myasthenia gravis (QMG) score was observed in 87% of participants (16.0% in the placebo group), with statistically significant difference in efficacy from the placebo group and manageable safety profile.

The ODD granted by EMA represents the recognition of telitacicept's innovative mechanism of action and potential in treating MG. RemeGen is proceeding the global multi-center phase III clinical trial of telitacicept in patients with MG to bring the breakthrough treatment option to more patients globally.

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SOURCE RemeGen Co., Ltd

FAQ

What is the significance of REGMY's telitacicept receiving EMA Orphan Drug Designation?

The designation provides RemeGen's telitacicept with protocol assistance, fee reductions, and 10 years of market exclusivity in Europe, accelerating development and approval processes for treating Myasthenia Gravis.

What were the Phase III clinical trial results for telitacicept in treating Myasthenia Gravis?

The trial showed 98.1% of participants achieved ≥3 points improvement in MG-ADL (vs 12% placebo) and 87% showed ≥5 points improvement in QMG score (vs 16% placebo), with manageable safety.

How does RemeGen's telitacicept work in treating Myasthenia Gravis?

Telitacicept blocks both BLyS and APRIL signaling pathways, inhibiting abnormally activated B cells and reducing pathogenic autoantibodies that attack neuromuscular junction proteins.

What is the current market status of REGMY's telitacicept?

Telitacicept is currently approved in China (May 2025) and is undergoing global multi-center phase III clinical trials for international expansion.

How common is Myasthenia Gravis, the condition that telitacicept treats?

Myasthenia Gravis affects 15-25 people per 100,000 globally, qualifying as a rare disease under EMA's definition of less than 5 in 10,000 in the EU.